An Observational, Multicenter Study to Evaluate the Use and Effectiveness of Doptelet® in Patients With ITP (ADOPT)

Sobi

Status: Active, not recruiting

Conditions: ITP; Immune Thrombocytopenia

Treatments: Drug: Avatrombopag

Study type: Observational

Funder types: Industry

Identifiers

NCT04943042

Sobi.Doptelet-001

Details and patient eligibility

About

This is a multi-center, observational, Phase 4 study in patients with Immune Thrombocytopenia (ITP) designed to describe the real-world effectiveness of Doptelet and assess the patterns of drug utilization to add to the knowledge base regarding the use of Doptelet in routine medical practice. Patients eligible for participation will, as part of their routine medical care, be receiving Doptelet for the treatment of ITP.

Full description

This is a multi-center, observational, Phase 4 study in patients with ITP designed to describe the real-world effectiveness of Doptelet and assess the patterns of drug utilization to add to the knowledge base regarding the use of Doptelet in routine medical practice.

Patients eligible for participation will, as part of their routine medical care, be receiving Doptelet for the treatment of ITP.

The scope of the study is to collect both retrospective and prospective data. The main part of the study will be prospective collecting data on usage, effectiveness, safety, patient- and clinician-reported outcomes and health economic parameters whereas the retrospective part will consist of collection of information on previous treatments, reason for treatment switch, healthcare resource use and, if applicable, Doptelet treatment prior to enrollment. The retrospective data collection will be based on the information available in the patient's medical records. Data will be collected for up to 12 months prior to Doptelet treatment start.

Prospective data will be collected at routine clinical visits throughout the study. Patients will be followed for 12 (+6) months and will be enrolled until their first scheduled visit after 12 months of enrollment, or until early termination, whichever occurs first.

Enrollment: 199 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Patient is ≥18 years of age
2. Established and well documented ITP diagnosis
3. Patient is treated with, or at enrollment prescribed, Doptelet for ITP. Decision to initiate treatment shall be made by the treating physician and independently from the decision to include the patient in the study
4. Signed and dated informed consent provided by the patient before any study-related activities are undertaken
5. Willing and able to comply with protocol requirements

Exclusion criteria

1. Enrollment in a concurrent clinical interventional study, or intake of an Investigational Medicinal Product (IMP), within three months prior to inclusion in this study
2. ITP secondary to Evan's syndrome, lupus and other autoimmune diseases
3. ITP secondary to other hematological disorders and hematological malignancies
4. ITP secondary to any other malignancies
5. ITP secondary to known drug toxicity
6. ITP secondary to any other disease considered relevant by the Investigator

Trial design

199 participants in 1 patient group

Full-analysis set (FAS)

Description:

The FAS includes all enrolled patients. The FAS will be used for all analyses.

Treatment:

Drug: Avatrombopag